A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

Phase 3

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: Placebo; Drug: TEZ/IVA; Drug: IVA

• Registration Number: NCT03559062
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

Detailed Description

Not available

Study Timeline

• Study Start: 2018-05-17
• Study First Submitted: 2018-06-05
• Study First Submitted QC: 2018-06-05
• Study First Posted: 2018-06-15
• Primary Completion: 2018-12-21
• Study Completion: 2018-12-21
• Results First Submitted: 2019-12-20
• Status Verified: 2020-02
• Results First Submitted QC: 2020-02-04
• Last Update Submitted: 2020-02-04
• Results First Posted: 2020-02-11
• Last Update Posted: 2020-02-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 67

Inclusion Criteria

• Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
• Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
• Participants with a screening LCI2.5 result ≥7.5.
• Participants who are able to swallow tablets.

Key

Exclusion Criteria

• Clinically significant cirrhosis with or without portal hypertension.
• Colonization with organisms associated with a more rapid decline in pulmonary status.
• Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.
TEZ/IVA	TEZ/IVA	Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
TEZ/IVA	IVA	Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
TEZ/IVA	Placebo	Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
Ivacaftor	IVA	Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
Ivacaftor	Placebo	Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.

Primary Outcome Measures

Name	Time	Method
Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8	From baseline through Week 8	LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

Secondary Outcome Measures

Name	Time	Method
Absolute Change in Sweat Chloride At Week 8	From baseline at Week 8	Sweat samples were collected using an approved collection device.
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8	From baseline through Week 8	The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit	From first dose of study drug up to safety follow-up visit (up to Week 12)

Trial Locations

Locations (27)

Hunter Medical Research Institute (HMRI); 🇦🇺 New Lambton Heights, Australia

Princess Margaret Hospital for Children; 🇦🇺 Perth, Australia

Lady Cilento Children's Hospital; 🇦🇺 South Brisbane, Australia

The Children's Hospital at Westmead; 🇦🇺 Westmead, Australia

Universitair Ziekenhuis Brussel - Campus Jette; 🇧🇪 Brussels, Belgium

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg; 🇧🇪 Leuven, Belgium

University of Copenhagen Rigshospitalet; 🇩🇰 Copenhagen, Denmark

Groupe Hospitalier Pellegrin - Hôpital des Enfants; 🇫🇷 Bordeaux Cedex, France

Hôpital Necker - Enfants Malades; 🇫🇷 Paris, France

Universitaetsklinikum Essen; 🇩🇪 Essen, Germany

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